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- ||||| Livmarli (maralixibat) / Mirum Pharma
Trial completion: A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (clinicaltrials.gov) - Oct 27, 2022
P3, N=93, Completed, Sponsor: Mirum Pharmaceuticals, Inc.
Active, not recruiting --> Completed
- Livmarli (maralixibat) / Mirum Pharma
REAL-WORLD SAFETY EXPERIENCE IN PATIENTS WITH ALAGILLE SYNDROME TREATED WITH MARALIXIBAT () - Oct 23, 2022 - Abstract #AASLD2022AASLD_2307;
MRX is well-tolerated in patients with ALGS in the real world setting through the EAP. GI events, FSV deficiency, nor liver enzyme elevations are treatment limiting in this setting, and ongoing surveillance continues.
- || Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Sep 19, 2022
P2, N=72, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
GI events, FSV deficiency, nor liver enzyme elevations are treatment limiting in this setting, and ongoing surveillance continues. Trial completion date: Aug 2024 --> Mar 2025 | Trial primary completion date: Feb 2023 --> Aug 2023
- | Livmarli (maralixibat) / Mirum Pharma, Takeda
New P3 trial: A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS) (clinicaltrials.gov) - Sep 16, 2022
P3, N=5, Not yet recruiting, Sponsor: Takeda
- | Livmarli (maralixibat) / Mirum Pharma, Takeda
New P3 trial: A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Sep 16, 2022
P3, N=9, Not yet recruiting, Sponsor: Takeda
- | Livmarli (maralixibat) / Mirum Pharma
Journal, HEOR: Maralixibat Treatment Response in Alagille Syndrome Is Associated with Improved Health-Related Quality of Life. (Pubmed Central) - Sep 14, 2022
Trial completion date: Aug 2024 --> Mar 2025 | Trial primary completion date: Feb 2023 --> Aug 2023 (s): The significant improvements in pruritus seen with maralixibat at week 48 of the ICONIC study are clinically meaningful and are associated with improved HRQoL.
- || Livmarli (maralixibat) / Mirum Pharma
P2 data, Review, Journal: Maralixibat for the treatment of PFIC: Long-term, IBAT inhibition in an open-label, Phase 2 study. (Pubmed Central) - Sep 3, 2022
The 7 responders survived with native liver and experienced clinically significant reductions in pruritus and meaningful improvements in growth and QoL. Maralixibat may represent a well-tolerated alternative to surgical intervention.
- ||| Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Aug 16, 2022
P3, N=90, Enrolling by invitation, Sponsor: Mirum Pharmaceuticals, Inc.
Maralixibat may represent a well-tolerated alternative to surgical intervention. Trial completion date: Dec 2022 --> Sep 2024 | Trial primary completion date: Nov 2022 --> Sep 2024
- || Livmarli (maralixibat) / Mirum Pharma
Clinical, Journal: Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases-Examination of cholestatic liver disease in Alagille syndrome. (Pubmed Central) - Jul 29, 2022
P=N/A, P2
In this investigation, inclusion and exclusion criteria from a published trial of maralixibat in Alagille syndrome (ALGS, ITCH NCT02057692) were applied to a prospective longitudinal cohort of children with cholestasis (LOGIC NCT00571272) to derive contextual comparator data for evolving clinical trials of intestinal bile acid transport inhibitors in ALGS...Weighted survival with native liver was 91% at 2 years in the ALGS NH. These investigations provide valuable real-world data that can serve as contextual comparators to current clinical trials, especially those without control populations, and highlight the value and importance of funded multicenter, prospective, natural history studies.
- | Livmarli (maralixibat) / Mirum Pharma
Journal: Impact of long-term administration of maralixibat on children with cholestasis secondary to Alagille syndrome. (Pubmed Central) - Jul 29, 2022
Maralixibat administration was associated with marked improvement in pruritus and QoL. Interpretation of these findings is complicated by the complex natural history of severe cholestasis in Alagille syndrome.
- | Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: RISE: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (clinicaltrials.gov) - Jul 19, 2022
P2, N=12, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Interpretation of these findings is complicated by the complex natural history of severe cholestasis in Alagille syndrome. Trial completion date: Jan 2023 --> Aug 2023 | Trial primary completion date: Jun 2022 --> Mar 2023
- ||||| Livmarli (maralixibat) / Mirum Pharma
Enrollment change: An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) (clinicaltrials.gov) - Jun 9, 2022
P3, N=93, Enrolling by invitation, Sponsor: Mirum Pharmaceuticals, Inc.
Trial completion date: Jan 2023 --> Aug 2023 | Trial primary completion date: Jun 2022 --> Mar 2023 N=30 --> 93
- ||||| Livmarli (maralixibat) / Mirum Pharma
Enrollment closed, Enrollment change: A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (clinicaltrials.gov) - Mar 23, 2022
P3, N=93, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
N=30 --> 93 Recruiting --> Active, not recruiting | N=30 --> 93
- volixibat (SAR 548304) / Mirum Pharma, Livmarli (maralixibat) / Mirum Pharma
Dosing ileal bile acid transporter inhibitors in the fasted state minimizes gastrointestinal adverse effects while maintaining pharmacodynamic effect (Poster Area) - Mar 16, 2022 - Abstract #EASLILC2022EASL_ILC_1152;
These data demonstrate that GI tolerability is improved when dosing an IBATi in the fasted state, versus dosing at mealtime or immediately after food intake. Animal data show that PD effect is maintained regardless of dosing time relative to mealtime, suggesting that efficacy can be maintained while minimizing GI events.
- || Livmarli (maralixibat) / Mirum Pharma
Review, Journal: Maralixibat: First Approval. (Pubmed Central) - Feb 19, 2022
Maralixibat is also under regulatory review for ALGS in Europe, and clinical development for cholestatic liver disorders including ALGS in patients under 1 year of age, PFIC and biliary atresia is continuing in several other countries. This article summarises the milestones in the development of maralixibat leading to this first approval for ALGS.
- || Livmarli (maralixibat) / Mirum Pharma
Clinical, Journal: Maralixibat relieves chronic cholestasis in children. (Pubmed Central) - Feb 19, 2022
This article summarises the milestones in the development of maralixibat leading to this first approval for ALGS. No abstract available
- | Livmarli (maralixibat) / Mirum Pharma
Journal: Maralixibat Chloride. (Pubmed Central) - Feb 10, 2022
No abstract available No abstract available
- ||| Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment closed, Enrollment change: MERGE: MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (clinicaltrials.gov) - Jan 20, 2022
P2, N=52, Active, not recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
No abstract available Enrolling by invitation --> Active, not recruiting | N=124 --> 52
- | Livmarli (maralixibat) / Mirum Pharma
Journal: Evaluation of chronic toxicity of cyclocreatine in beagle dogs after oral gavage administration for up to 23 weeks. (Pubmed Central) - Jan 1, 2022
Cyclocreatine (LUM-001) was evaluated for chronic toxicity (23 weeks) in beagle dogs to support clinical development in patients with creatine transporter deficiency (CTD) disorder...Cyclocreatine was absorbed rapidly with mean T within 1 to 2 h and half-life ranged between 2.17 and 2.79 h on Day 1, however, on the final day of dosing, it ranged between 5.80 and 8.77 h (males) and 10.3 to 13.1 h (females). To conclude, in this study the lungs, kidneys, heart, skeletal and smooth muscles were identified as the target organs of cyclocreatine toxicity in beagle dogs.
- || Livmarli (maralixibat) / Mirum Pharma
Clinical, P2 data, Journal: Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. (Pubmed Central) - Dec 23, 2021
P2
In children with Alagille syndrome, maralixibat is, to our knowledge, the first agent to show durable and clinically meaningful improvements in cholestasis. Maralixibat might represent a new treatment paradigm for chronic cholestasis in Alagille syndrome.
- | Livmarli (maralixibat) / Mirum Pharma
Trial primary completion date: A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (clinicaltrials.gov) - Dec 10, 2021
P3, N=30, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Maralixibat might represent a new treatment paradigm for chronic cholestasis in Alagille syndrome. Trial primary completion date: Oct 2021 --> Jul 2022
- Livmarli (maralixibat) / Mirum Pharma
[VIRTUAL] PREDICTORS OF 6-YEAR EVENT-FREE SURVIVAL IN PATIENTS WITH ALAGILLE SYNDROME TREATED WITH MARALIXIBAT, AN IBAT INHIBITOR () - Nov 2, 2021 - Abstract #AASLD2021AASLD_2418;
Week 48 TB and sBA levels, as well as improvement in pruritus and age of initiation of MRX, were associated with lower long-term rates of clinically important events and transplantation. These data identify potential prognostic markers which may help guide clinical decisions in patients with ALGS treated with MRX.
- Livmarli (maralixibat) / Mirum Pharma
[VIRTUAL] APPLICATION OF REAL-WORLD EVIDENCE ANALYTICS: A 6-YEAR EVENT-FREE SURVIVAL ANALYSIS IN ALAGILLE SYNDROME OF THE GALA CLINICAL RESEARCH DATABASE AND MARALIXIBAT TREATED PATIENTS () - Nov 2, 2021 - Abstract #AASLD2021AASLD_2400;
This RWE analysis provides a potential method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible. Limitations will always be present given lack of prospective conduct and inherent biases, though sensitivity analyses can help mitigate and aid interpretation.
- Livmarli (maralixibat) / Mirum Pharma
[VIRTUAL] UTILIZATION OF MULTI-CENTER PROSPECTIVE LONGITUDINAL DATABASES TO INFORM CLINICAL TRIALS IN RARE DISEASES – EXAMINATION OF CHOLESTATIC LIVER DISEASE IN ALAGILLE SYNDROME (ALGS) () - Oct 21, 2021 - Abstract #AASLD2021AASLD_2208;
P=N/A, P2
This cohort demonstrated a significant improvement in pruritus, a fall in platelet count and increase in height in ALGS during routine clinical follow up using currently available therapeutic agents . This clinical data may be useful in contextualizing the findings of clinical trials.
- | Livmarli (maralixibat) / Mirum Pharma
Trial completion date: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Sep 28, 2021
P2, N=72, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
This clinical data may be useful in contextualizing the findings of clinical trials. Trial completion date: Aug 2025 --> Aug 2024
- || Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Sep 20, 2021
P2, N=72, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Trial completion date: Aug 2025 --> Aug 2024 Trial completion date: Apr 2023 --> Aug 2025 | Trial primary completion date: Nov 2022 --> Feb 2023
- | Livmarli (maralixibat) / Mirum Pharma
Trial initiation date, Trial primary completion date: RISE: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (clinicaltrials.gov) - Sep 17, 2021
P2, N=12, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Trial completion date: Apr 2023 --> Aug 2025 | Trial primary completion date: Nov 2022 --> Feb 2023 Initiation date: Mar 2021 --> Sep 2021 | Trial primary completion date: Jan 2023 --> Jun 2022
- | maralixibat (SHP625) / Mirum Pharma
One In, One Out: Mirum Seeks New EU Indication For Maralixibat, Withdraws Another https://t.co/7Pvc1KV3ta #PinkSheet (Twitter) - Sep 15, 2021
- | Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (clinicaltrials.gov) - Sep 14, 2021
P3, N=30, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Initiation date: Mar 2021 --> Sep 2021 | Trial primary completion date: Jan 2023 --> Jun 2022 Trial completion date: Jul 2021 --> May 2022 | Trial primary completion date: Jul 2021 --> Oct 2021
- || maralixibat (SHP625) / Mirum Pharma, odevixibat (A4250) / Albireo
Review, Journal: Ileal bile acid transporter inhibition as an anticholestatic therapeutic target in biliary atresia and other cholestatic disorders. (Pubmed Central) - Aug 18, 2021
By reducing return of bile acids to the cholestatic liver, IBAT inhibitors may potentially lessen or delay liver damage associated with the hepatotoxicity and cholangiopathy of bile acid accumulation. The clinical programs of 2 IBAT inhibitors in development for the treatment of pediatric cholestatic liver diseases, maralixibat and odevixibat, are highlighted.
- | maralixibat (SHP625) / Mirum Pharma
Preclinical, Journal: Evaluation of chronic toxicity of cyclocreatine, a creatine analog, in Sprague Dawley rat after oral gavage administration for up to 26 weeks. (Pubmed Central) - Jul 10, 2021
Cyclocreatine (LUM-001), a creatine analog, was evaluated for its nonclinical toxicity in Sprague Dawley (SD) rats...In conclusion, chronic administration of cyclocreatine by oral gavage in Sprague Dawley rats induced the seizures and microscopic lesions in the brain, testes and thyroid. Based on the results of this study the highest tested dose of 600 mg/kg/day (mean C of 151.5 μg/mL; AUC of 1970 h*μg/mL) was considered the maximum tolerated dose (MTD) in SD rats.
- ||| Livmarli (maralixibat) / Mirum Pharma, Takeda
Enrollment change, Trial completion date, Trial primary completion date: MERGE: MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (clinicaltrials.gov) - Jul 1, 2021
P2, N=124, Enrolling by invitation, Sponsor: Mirum Pharmaceuticals, Inc.
Based on the results of this study the highest tested dose of 600 mg/kg/day (mean C of 151.5 μg/mL; AUC of 1970 h*μg/mL) was considered the maximum tolerated dose (MTD) in SD rats. N=54 --> 124 | Trial completion date: Jan 2023 --> Dec 2023 | Trial primary completion date: Dec 2022 --> Dec 2023
- || Livmarli (maralixibat) / Mirum Pharma
Enrollment open: RISE: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (clinicaltrials.gov) - May 10, 2021
P2, N=12, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
N=54 --> 124 | Trial completion date: Jan 2023 --> Dec 2023 | Trial primary completion date: Dec 2022 --> Dec 2023 Not yet recruiting --> Recruiting
- maralixibat (SHP625) / Mirum Pharma
[VIRTUAL] An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome (Poster area) - Apr 9, 2021 - Abstract #EASLILC2021EASL_ILC_1025;
Diarrhea and abdominal pain were the most common AEs, were mostly transient in nature, and resolved without any action taken. ALT rises were not associated with clinical sequelae and appeared to be consistent with the natural history of the disease.
- ||| maralixibat (SHP625) / Mirum Pharma
FDA event, NDA, PDUFA date, Review: $MRIM Announces FDA Acceptance of NDA and Priority Review for Maralixibat in Alagille Syndrome PDUFA 09/29/21. Adcomm is not currently planned. (Twitter) - Mar 29, 2021
- rifampicin / Generic mfg., sertraline / Generic mfg., bezafibrate / Generic mfg.
[VIRTUAL] PHARMACOLOGICAL THERAPY OF PRURITUS IN PRIMARY BILIARY CHOLANGITIS: A SYSTEMATIC REVIEW AND META-ANALYSIS (DDW Virtual) - Mar 15, 2021 - Abstract #DDW2021DDW_2760;
The effect size on the rifampin trial is limited due to its small sample size. Further studies are required to identify better therapeutic options for this debilitating condition.
- ||| Livmarli (maralixibat) / Mirum Pharma
Enrollment open, Trial initiation date: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Feb 21, 2021
P2, N=72, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Further studies are required to identify better therapeutic options for this debilitating condition. Not yet recruiting --> Recruiting | Initiation date: Dec 2020 --> Mar 2021
- ||| maralixibat (SHP625) / Mirum Pharma
NDA: $MIRM Announces Completion of Rolling NDA Submission for Maralixibat in Alagille Syndrome (Twitter) - Feb 1, 2021
- || Livmarli (maralixibat) / Mirum Pharma
New P2 trial: RISE: A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (clinicaltrials.gov) - Jan 27, 2021
P2, N=12, Not yet recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
- || maralixibat (SHP625) / Mirum Pharma
Clinical, European regulatory: $MIRM Announces EMA Validation of the Marketing Authorization Application for Maralixibat in Patients with PFIC2 (Twitter) - Nov 30, 2020
- | Livmarli (maralixibat) / Mirum Pharma
Trial completion date, Trial primary completion date: A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (clinicaltrials.gov) - Nov 23, 2020
P3, N=30, Recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
Not yet recruiting --> Recruiting | Initiation date: Dec 2020 --> Mar 2021 Trial completion date: Dec 2020 --> Jul 2021 | Trial primary completion date: Dec 2020 --> Jul 2021
- maralixibat (SHP625) / Mirum Pharma
[VIRTUAL] PRELIMINARY ANALYSIS OF ITCH AND IMAGINE II – OUTCOME OF LONG-TERM ADMINISTRATION OF MARALIXIBAT IN CHILDREN WITH ALAGILLE SYNDROME () - Nov 2, 2020 - Abstract #AASLD2020AASLD_2147;
P2
These changes persisted for at least 96 weeks in those who were able to continue to receive MRX. The clinical significance of increased ALT and SAEs with MRX administration is uncertain in the context of the natural history of ALGS.
- | maralixibat (SHP625) / Mirum Pharma
Es muy duro lidiar con el prurito cuando tu hijo tiene #sindromedealagille dormir es imposible... @mirumpharma ha desarrollado #maralixibat un medicamento que ha probado ser seguro y eficiente disminuyendo el prurito 😃. Gracias 🙏🏻 @ESPGHANelearn @VertexPharma @RetrophinRare (Twitter) - Oct 19, 2020
- maralixibat (SHP625) / Mirum Pharma
[VIRTUAL] NATURAL VARIABILITY OF PRURITUS IN ALAGILLE SYNDROME; AN ANALYSIS FROM THE ICONIC STUDY UTILIZING THE ITCH REPORTED OUTCOME OBSERVER (ITCHRO [OBS]) TOOL () - Oct 11, 2020 - Abstract #AASLD2020AASLD_1792;
This analysis of pruritus severity and frequency during the screening period from the maralixibat ICONIC study reveals that itching in children with ALGS is persistent over time, rather than intermittent, in both intensity and frequency. The ICONIC study is an ongoing clinical trial with participants receiving over four years of maralixibat treatment; recently presented data demonstrate control of cholestasis and pruritus associated with ALGS.
- maralixibat (SHP625) / Mirum Pharma
[VIRTUAL] PRURITUS INTENSITY IS ASSOCIATED WITH CHOLESTASIS BIOMARKERS AND QUALITY OF LIFE MEASURES AFTER MARALIXIBAT TREATMENT IN CHILDREN WITH ALAGILLE SYNDROME () - Oct 11, 2020 - Abstract #AASLD2020AASLD_341;
Pruritus was significantly correlated with biomarkers of cholestasis at 48 weeks of MRX treatment including sBA, ATX, CSS score, specific PedsQL measures and sBA subspecies. Further work on sBA subspecies is warranted to further understand mechanisms of cholestatic pruritus and the positive treatment effects of MRX.
- || maralixibat (SHP625) / Mirum Pharma
👏🏻Enhorabuena @aelmhu por sumar a @mariancorral a esta causa #enfermedadesraras #medicamentoshuerfanos #sindromedealagille #maralixibat @mirumpharma @dgenesmurcia @FEDER_ONG @eurordis @TransplantChild (Twitter) - Sep 9, 2020
- |||||| Livmarli (maralixibat) / Mirum Pharma
New trial: A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS) (clinicaltrials.gov) - Aug 28, 2020
P, N=0, Available, Sponsor: Mirum Pharmaceuticals, Inc.
- |||| Livmarli (maralixibat) / Mirum Pharma
New P2 trial: EMBARK: Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (clinicaltrials.gov) - Aug 23, 2020
P2, N=72, Not yet recruiting, Sponsor: Mirum Pharmaceuticals, Inc.
- || A3384 / Albireo, maralixibat (SHP625) / Mirum Pharma, Ocaliva (obeticholic acid) / Intercept
Clinical, Review, Journal: Adjuvant treatments for biliary atresia. (Pubmed Central) - Aug 11, 2020
Newer modalities such as antiviral therapy (AVT), immunoglobulin, FXR agonists (e.g., obeticholic acid), ileal bile acid transporter (IBAT) antagonists (e.g., maralixibat) remain unproven. This article reviews the current evidence for the efficacy of adjuvant medical therapy in BA.
- || maralixibat (SHP625) / Mirum Pharma
Biomarker, Clinical, Journal: Unraveling the Relationship Between Itching, Scratch Scales, and Biomarkers in Children With Alagille Syndrome. (Pubmed Central) - Jul 7, 2020
In a previously reported trial of maralixibat, an investigational antipruritic agent, itching was assessed using a digital diary based on twice-daily caregiver observation of itching severity (Itch Reported Outcome, ItchRO[Observer])...Hypothesis-generating analyses implicate sleep disturbance and fatigue as key associations with caregiver observations of itching. This is highly relevant to the selection of surrogate endpoints for clinical trials of pruritus therapies.